Going against the advice of its own advisors, the European Commission has said that PTC Therapeutics’ Translarna therapy for Duchenne muscular dystrophy (DMD) should stay
The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to paus
Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD), becoming the first non-steroidal option for
Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy has failed to meet its primary objective in a phase 3 trial, but the company says it will file to extend
Swiss biotech Santhera is on the brink of its first regulatory approval for Duchenne muscular dystrophy treatment vamorolone, after getting a positive opinion on the drug
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.